Despite advancements, most rare diseases lack effective therapies, underscoring the necessity for a strategic overhaul in the approach to research and development. To address the multifaceted challenges in rare disease research, a unified framework that encompasses the recommendations across clinical trials, diagnostics, and translational research is proposed by the Rare Disease Moonshot. Central to this […]
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The Rare Disease (RD) Moonshot steering group has just published its recommendations for research funders and health research players from the industry and public sector on research needs that can be best addressed by public-private collaborations to optimise clinical trials in small populations. The RD Moonshot was set up to boost public private collaborations in the […]
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